Practice Variations in Managing Infantile Hemangiomas.

Infantile hemangioma (IH) is the most common benign tumor of infancy. For children with IH who require treatment, propranolol and other beta blockers have been shown to be safe and effective. Although consensus guidelines for managing IH have been published, anecdotal experience suggests that there remain variations in management. This study was performed to document these variations amongst providers and to identify areas for future research. We conducted an Internet-based survey of clinicians who treat patients with IH. Hypothetical cases and management scenarios were presented. Twenty-nine respondents participated in the survey. Most respondents use generic propranolol in infants with growing IH of the head and neck, with a goal dose of 2 mg/kg/d, until ~1 year of age. A variety of management strategies were documented including which patients should be treated, optimal dose and duration of therapy, how patients should be monitored, which patients should get additional workup, how propranolol should best be discontinued, and how often to see patients in follow-up. This study demonstrates wide practice variations in managing patients with IH. Further research is indicated to address these variations and develop additional/updated evidence-based guidelines.

Vascular Anomalies Care in the United States: A Cross-Sectional National Survey.

OBJECTIVE: To characterize the current distribution, composition, and practice patterns of multidisciplinary vascular anomalies (VAs) teams in the US. STUDY DESIGN: This is a cross-sectional survey of children's hospitals in the US offering VAs care. We approached 142 children's hospitals that provided care for VAs via email. The survey evaluated VA clinic location, medical staffing, research participation, and treatments offered. The survey was administered between October 2021 and July 2022. RESULTS: Participants from 95 eligible hospitals responded to the survey (response rate = 67%). Large areas of the Midwest and Northwest US had no available multidisciplinary VA teams or clinics. Most respondents worked at academic centers (89%), with 66% at a freestanding children's hospital, and 56% reported having a multidisciplinary clinic. Most common physician participants in clinic included hematology-oncology (91%), interventional radiology (87%), dermatology (85%), plastic surgery (81%), and otolaryngology (74%). Only 38% of programs included medical geneticists. Smaller hospitals had fewer medical and ancillary staff and offered fewer therapeutic options. Research was available at most larger institutions (69%) but less commonly at smaller hospitals (34%). CONCLUSIONS: Major portions of the US lack multidisciplinary VA care. Furthermore, VA programs vary in composition and geneticists are absent from the majority of programs. These findings should inform efforts to address disparate access and develop standards of care for multidisciplinary VA care in the US.

Microvascular volume visualization utilizing computed tomographic angiography data facilitates resection of a complicated massive congenital hemangioma.

CT angiography (CTA) can be used for planning procedural and operative therapies for neonatal vascular lesions, such as congenital hemangiomas (CH), that are too morbid for medical therapy. Neonatal anatomy can be displayed within a small enough field-of-view that the nominal resolution of a modern CT scanner can be realized with a standard 512 × 512 storage matrix, yielding isometric ultrahigh resolution data and allowing for microvascular volume visualization. This case report details the creation and use of microvascular volume visualizations of CTA data during pre-procedural planning for treatment of this pathological entity.

Small cell carcinoma of the ovary of hypercalcemic type: a case report.

The authors report a case of small cell carcinoma of the ovary, hypercalcemic type (SCCOHT), in a mother and daughter and discuss the possibility of a heritable risk. Both mother and daughter were treated at the same institution for SCCOHT. A 23-year-old woman presented with hypercalcemia 4 months after giving birth to her daughter. She was diagnosed as having SCCOHT. Despite surgery, chemotherapy, and radiation, she died of the disease 11 months after diagnosis. Eleven years later, her daughter presented with a histologically and immunophenotypically identical SCCOHT tumor. She received postoperative chemotherapy and radiation but, eventually, relapsed and died of the disease at 27 months after the initial diagnosis. Small cell carcinoma of the ovary, hypercalcemic type, is an uncommon and aggressive malignancy that occurs in young women, which is associated with a solid ovarian tumor and hypercalcemia. Despite aggressive multimodality treatment, most patients die within 2 years of diagnosis. Genetic counseling, sonographic ovarian surveillance and serum calcium monitoring at early age, and even prophylactic oophorectomy should be considered for surviving at-risk family members.

Successful treatment of ifosfamide neurotoxicity with dexmedetomidine.

Neurotoxicity related to the use of ifosfamide is a well-known complication. While the use of methylene blue is a known antidote, symptomatic treatment of the central nervous system (CNS) effects can be challenging. We present a case of class IV neurotoxicity with the successful treatment of symptomology. In this case report we present a 2-year-old female with relapsed alveolar rhabdomyosarcoma undergoing palliative chemotherapy. Patient received ifosfamide in addition to etoposide and mesna. The patient developed acute hallucinations, agitation, and delirium. The patient was transferred to the pediatric intensive care unit where she was administered dexmedetomidine overnight in addition to methylene blue. The patient awoke the next morning following discontinuation of the dexmedetomidine infustion and subsequently had no further central nervous system effects. This case demonstrates the novel use of an alpha-2 agonist in the treatment of neurotoxicity related to ifosfamide administration.

Lack of circulating megakaryoblasts in newborn peripheral blood: development and validation of a sensitive flow cytometric detection method.

It is currently thought that approximately 1% of children with Down syndrome will develop a "premalignant" syndrome known as transient myeloproliferative disorder (TMD). Prospective, population-based studies of the incidence of TMD in Down syndrome infants is lacking. Although most cases of TMD resolve by 1 year of age, data suggest that 10% to 20% of Down syndrome patients with TMD develop AML-M7 (megakaryoblastic leukemia). To identify the true incidence of TMD in the Down syndrome population, a sensitive, rapid, and cost-effective method of quantifying circulating megakaryoblasts in large numbers of patients was needed. In this pilot study, the authors tested the hypothesis that there are fewer than 1% megakaryoblasts of nucleated cells circulating in the blood of normosomic infants. Four-antigen flow cytometry was used to establish the percentage of megakaryoblasts present in each of 100 cord blood samples collected blindly from "normosomic" live births. There was a mean percentage of 0.017% megakaryoblasts in 100 cord blood samples from normosomic infants. Flow cytometry proved to be a sensitive, rapid, and reproducible method for the quantification of megakaryoblasts. Less than 1% of circulating nucleated cells in the blood of newborn infants are megakaryoblasts, providing a comparison population for the authors' larger proposed incidence study.

Atypical presentation and progression of glioblastoma multiforme in a 6-year-old girl: multidisciplinary case report.

Glioblastoma multiforme is the most common adult malignant brain tumor but is notably less common in children. The authors describe the case of a child who presented for evaluation and treatment of neurologic signs caused by a brain stem glioma. Response to radiotherapy and chemotherapy with temozolomide was initially positive, but later extensive leptomeningeal metastasis developed. Biopsy proved the lesion to be glioblastoma multiforme. During salvage irradiation to the spine and unirradiated brain, the patient complained of hip and femur pain. Subsequent radiographs demonstrated multiple bony metastases. This pattern of spread is uncharacteristic and emphasizes the importance of adequate metastatic evaluation.

Infections diagnosed in the first year after pediatric stem cell transplantation.

BACKGROUND: Cumulative incidence of infections in the first year posttransplantation in adult patients has been well-described. Such description is less than complete for pediatric stem cell transplantation (SCT) patients. Further among those patients who have been infected, analysis of risk factors for infection has not been well-described for a large cohort of pediatric SCT patients. METHODS: We conducted a retrospective cohort study of infections in the first year after SCT at Duke University Medical Center. We recorded all infections in the first year after transplantation. We determined incidences for 6 categories of infection: gram-negative rods; gram-positive cocci; yeast species; Aspergillus sp.; adenovirus; and cytomegalovirus. We determined incidences based on type of transplant and days post transplantation. We also completed bivariable and multivariable analysis of risk factors [neutropenia, graft vs. host disease (GVHD) and GVHD treatment] for infection type among those children who were infected. RESULTS: We evaluated 510 transplants in 485 children. There were 584 infections in the first year after transplantation. During the first 30 days posttransplantation, type of transplantation did not predict incidence of infection or type of infection. After 30 days children who received unrelated cord blood transplant and matched unrelated donor transplant were at much higher risk of infection than were patients who received autologous, matched sibling or haploidentical transplant (P < 0.001). Patients who received unrelated cord blood or matched unrelated donor transplantation were at higher risk of aspergillosis (P = 0.002), candidiasis (P = 0.005) and adenovirus (P < 0.0001) but not cytomegalovirus (P = 0.18). In analysis of risk factors among those infected, patients with aspergillosis were more likely to have severe GVHD: multivariable 1 year risk ratio, 7.5; 95% confidence interval, 3.0, 18.4. Neutropenia was more strongly associated with gram-negative rod infection than any other type of infection. CONCLUSIONS: The incidence of infection immediately after transplantation did not differ significantly by type of transplant in this pediatric population. Type of transplant predicted increased incidence of infection 30 days posttransplantation and increased incidence of infection with several organisms traditionally associated with a high mortality rate in the transplant population.